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PubMed Journals Articles About "Affordable Sickle Cell Disease Gene Therapies" RSS

17:12 EST 10th December 2019 | BioPortfolio

Affordable Sickle Cell Disease Gene Therapies PubMed articles on BioPortfolio. Our PubMed references draw on over 21 million records from the medical literature. Here you can see the latest Affordable Sickle Cell Disease Gene Therapies articles that have been published worldwide.

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Showing "Affordable sickle cell disease gene therapies" PubMed Articles 1–25 of 51,000+

Multicenter Evaluation of HemoTypeSC as a Point-of-Care Sickle Cell Disease Rapid Diagnostic Test for Newborns and Adults Across India.

Sickle cell anemia is the commonest genetic disorder in India, and the frequency of the sickle cell gene is very high in the remote tribal areas where facilities are generally limited. Therefore, a rapid and affordable point-of-care test for sickle cell disease is needed.


Hematopoietic stem cell transplantation and cellular therapy in sickle cell disease: where are we now?

Sickle cell disease (SCD) is a common monogenic disorder that is characterized by an A to T substitution in the β-globin gene that leads to the production of hemoglobin S (HbS). Polymerization of HbS leads to significant morbidity including vaso-occlusion, pain, hemolytic anemia, and end organ damage. Allogeneic hematopoietic cell transplantation (allo-HCT) is the only curative treatment; however, suitable donors are not always readily available. This study reviews the current status of allo-HCT and autolo...

Generation and characterization of induced pluripotent stem cell line (IGIBi001-A) from a sickle cell anemia patient with homozygous β-globin mutation.

Sickle cell disease (SCD) is an autosomal recessive disorder caused by a mutation in β-globin (HBB) gene. We have generated an induced pluripotent stem cell (iPSC) line, IGIBi001-A from an Indian sickle cell patient with a homozygous HBB gene mutation using Sendai virus reprogramming system. Characterization of IGIBi001-A showed that these iPSCs are transgene-free and expressed pluripotent stem cell markers. They had a normal karyotype and were able to differentiate into all three germ layers. This new SCD...


A Review of Automated Methods for the Detection of Sickle Cell Disease.

Detection of sickle cell disease is a crucial job in Medical Image Analysis. It emphasizes elaborate analysis of proper disease diagnosis after accurate detection followed by a classification of irregularities, which plays a vital role in the sickle Cell disease diagnosis, treatment planning, and treatment outcome evaluation. Proper segmentation of complex cell clusters makes sickle cell detection more accurate and robust. Cell morphology has a key role in the detection of the sickle cell because the shapes...

Preference-based measure of health-related quality of life and its determinants in sickle cell disease in Nigeria.

Health-related quality of life (HRQL) and economic burden are important issues for people with sickle cell disease (SCD) owing to better survival due to medical advances. Preference-based or utility information is necessary to make informed economic decisions on treatment and alternative therapies. This study aimed to assess preference-based measures of HRQL in sickle cell patients.

Association of the Δ Mutant Genotype with Sickle Cell Disease in Egyptian Patients.

Sickle cell disease is considered the most common single base mutation in the world, with >250,000 new patients being discovered each year. It consists of a wide spectrum of clinical presentations and complications. The is the mutant genotype of C-C chemokine receptor 5 (CCR5). It is widely distributed due to several micro organisms that target macrophages in different populations. Theoretically, confers an advantage to sickle cell disease patients. The chronic inflammatory response is the main pathogenes...

Prevalence and factors associated with renal dysfunction among children with sickle cell disease attending the sickle cell disease clinic at a tertiary hospital in Northwestern Tanzania.

Little is known on how the interaction between Sickle Cell Disease (SCD) and renal insults caused by other coexisting conditions in Sub Saharan Africa such as urinary schistosomiasis, malnutrition and HIV affect the prevalence of renal dysfunction in children with SCD.

Epidemiological, clinical, and severity characterization of sickle cell disease in a population from the Brazilian Amazon.

Sickle cell disease (SCD) is a chronic inflammatory condition caused by a point mutation in the HBB gene. Here we characterized the clinical presentation of SCD in a population from Amazonas State in northern Brazil, in order to evaluate whether the higher Amerindian ancestry observed in this relatively isolated geographic region would influence the clinical presentation of SCD.

Subperiosteal Orbital Hematoma: Imaging Findings of a Rare Complication of Sickle Cell Disease: Subperiosteal orbital hematoma is a rare entity mainly seen in pediatric patients with sickle cell disease and occurs secondary to local vascular disturbances following facial bone infarction.

Sickle cell disease is the most common hemoglobinopathy. Homozygous patients are prone to vaso-occlusive crises. A 19-year-old male patient with the homozygous sickle cell trait was admitted to the hospital due to a sickle cell crisis. During his admission he developed a left periorbital edema. The diagnosis of a subperiosteal orbital hematoma (SOH) was made by CT and MRI imaging. SOH is a rare complication of a VOC. The clinical course is mostly self-resolving, with some cases reporting the need for surgic...

Pediatric to Adult Transition in Sickle Cell Disease: Survey Results from Young Adult Patients.

We surveyed sickle cell disease (SCD) patients who transitioned from pediatric care at Texas Children's Hematology Center (TCHC) to adult care to determine the characteristics of patients with an adult SCD provider, continuation rates of pre-transition therapies, and patient perceptions of the transition process.

The increased neopterin content in turkish pediatric patients with sickle cell anemia.

In the present study, the possible activation of cellular immunity in SCD patients was investigated. As immune activation parameters, neopterin concentrations and kynurenine/tryptophan ratio for tryptophan degradation in 35 pediatric patients with sickle cell disease (31 HbSS and 4 HbSß) were determined. Our results have shown that neopterin levels (both urinary and serum) are increased in pediatric patients with sickle cell disease. The increase in neopterin concentration was accompanied by significantly ...

Association of sickle cell trait with atrial fibrillation: The REGARDS cohort.

Sickle cell trait (SCT), sickle cell disease's (SCD) carrier status, has been recently associated with worse cardiovascular and renal outcomes. An increased prevalence of atrial fibrillation (AF) is documented in SCD patients; however, studies in individuals with SCT are lacking.

Prevalence of enuresis and its impact in quality of life of patients with sickle cell disease.

Evidence indicates an increase in the prevalence of enuresis in individuals with sickle cell disease. The present study aims to evaluate the prevalence and impact of enuresis on quality of life in individuals with sickle cell disease.

The Risk of Pregnancy Related Hypertension Disorder Associated with Sickle Cell Trait in U.S. Service Women.

The health impact of the heterozygous (Hb Ss) Sickle Cell Trait (SCT) among the estimated one million females of reproductive age in the United States on pregnancy related hypertensive disorders (PRHD) including preeclampsia is not as well understood or researched as Sickle Cell Disease (SCD).

American Society of Hematology 2019 guidelines for sickle cell disease: cardiopulmonary and kidney disease.

Prevention and management of end-organ disease represent major challenges facing providers of children and adults with sickle cell disease (SCD). Uncertainty and variability in the screening, diagnosis, and management of cardiopulmonary and renal complications in SCD lead to varying outcomes for affected individuals.

Highly efficient editing of the β-globin gene in patient-derived hematopoietic stem and progenitor cells to treat sickle cell disease.

Sickle cell disease (SCD) is a monogenic disorder that affects millions worldwide. Allogeneic hematopoietic stem cell transplantation is the only available cure. Here, we demonstrate the use of CRISPR/Cas9 and a short single-stranded oligonucleotide template to correct the sickle mutation in the β-globin gene in hematopoietic stem and progenitor cells (HSPCs) from peripheral blood or bone marrow of patients with SCD, with 24.5 ± 7.6% efficiency without selection. Erythrocytes derived from gene-edited cell...

Exploring the Needs of Adolescents With Sickle Cell Disease to Inform a Digital Self-Management and Transitional Care Program: Qualitative Study.

Accessible self-management interventions are critical for adolescents with sickle cell disease to better cope with their disease, improve health outcomes and health-related quality of life, and promote successful transition to adult health care services. However, very few comprehensive self-management and transitional care programs have been developed and tested in this population. Internet and mobile phone technologies can improve accessibility and acceptability of interventions to promote disease self-man...

Acute Care Utilization at End of Life in Sickle Cell Disease: Highlighting the Need for a Palliative Approach.

People with sickle cell disease (SCD) have a life expectancy of

Splenectomy is not associated with a higher tricuspid regurgitant jet velocity in people with sickle cell anemia.

Vascular complications such as pulmonary hypertension (PH) occur at an increased rate following splenectomy in patients with various hemolytic blood disorders including thalassemia. The goal of this retrospective cross-sectional analysis was to assess the independent association of splenectomy with an elevated tricuspid regurgitation velocity (TRV) in people with homozygous sickle cell disease (HbSS). TRV is a noninvasive screening test for PH and a surrogate marker of prognosis in sickle cell disease (SCD)...

Combined genetic disruption of K-Cl cotransporters and Gardos channel KCNN4 rescues erythrocyte dehydration in the SAD mouse model of sickle cell disease.

Excessive red cell dehydration contributes to the pathophysiology of sickle cell disease (SCD). The densest fraction of sickle red cells (with the highest corpuscular hemoglobin concentration) undergoes the most rapid polymerization of deoxy-hemoglobin S, leading to accelerated cell sickling and increased susceptibility to endothelial activation, red cell adhesion, and vaso-occlusion. Increasing red cell volume in order to decrease red cell density can thus serve as an adjunct therapeutic goal in SCD. Regul...

Asthma in children with sickle cell disease.

Asthma is common in children with sickle cell disease (SCD) and appears to be associated with increased morbidity. Providers caring for children with SCD have struggled with the question of whether asthma exists as a true comorbidity or whether certain aspects of the chronic inflammatory disease gives children with SCD an asthma-like phenotype.

Decreased Bleeding Incidence with Direct Oral Anticoagulants Compared to Vitamin K Antagonist and Low-Molecular-Weight Heparin in Patients with Sickle Cell Disease and Venous Thromboembolism.

Venous thromboembolism (VTE) is a recognized complication of sickle cell disease (SCD), yet the optimal pharmacologic anticoagulant is unknown.

Sickle cell disease complications: Prevalence and resource utilization.

This study evaluated the prevalence rate of vaso-occlusive crisis (VOC) episodes, rates of uncomplicated and complicated VOC episodes, and the primary reasons for emergency room (ER) visits and inpatient admissions for sickle cell disease (SCD) patients.

Reflections of Healthcare Experiences of African Americans With Sickle Cell Disease or Cancer: A Qualitative Study.

The experiences of African American adult patients before, during, and after acute care utilization are not well characterized for individuals with sickle cell disease (SCD) or cancer.

Current and emerging treatments for sickle cell disease.

Sickle cell disease (SCD) is a group of inherited blood disorders affecting the hemoglobin, shortening the lifespan of erythrocytes, and causing them to take on a distinctive sickled shape that can lead to vaso-occlusion. Current treatment aims to reduce morbidity and mortality through hydroxyurea, erythrocyte transfusion, and hematopoietic stem cell transplantation. This article reviews the disease process, typical presentations, complications, and acute and chronic treatment options.


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